Abstract
Whether new versions of currently marketed, or de novo biological drugs, biobetters are products specifically designed to maximize clinical performance. Performance optimization may include, among other improvements, enhanced targeting and delivery, increased activity and half-life, and reduced immunogenicity. Thus, it is critical that such products are developed efficiently and quickly, to allow patients access to drugs that provide maximum clinical benefits.
Over the past 30 years, legislative mandates provided FDA with the flexibility to offer incentive programs and define novel regulatory pathways to aid in drug development, particularly for drugs to treat serious or life threatening diseases or unmet medical needs. These programs have been crucial for the approval of, for example, antiviral therapies for AIDS, or orphan drugs to treat rare diseases. This chapter outlines the salient features of several expedited regulatory pathways and selected incentive programs, provides examples of “biobettering” strategies and describes how expedited regulatory pathways and incentive program may be applied to the development of biobetter drugs.
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References
Best Pharmaceuticals for Children Act (2002) Pub. L. 107–109 Reauthorized in 2007 by Pub. L. 110–85 (Food and Drug Administration Amendments Act), and permanently reauthorized in 2012 by Pub. L. 112–114 (Food and Drug Administration Safety and Innovation Act)
Davis PB (2011) Therapy for cystic fibrosis – the end of the beginning? N Engl J Med 365:1734–1735
Food and Drug Administration Modernization Act (1997) Pub. L. 105–115. 111 Stat. 2036. Section 111. Pediatric studies of drugs
Federal Food, Drug, and Cosmetic Act (2012a) 21 U.S.C. Section 355a: pediatric studies of drugs
Federal Food, Drug, and Cosmetic Act (2012b) 21 U.S.C. Section 355n-1: priority review for qualified infectious disease products
Federal Food, Drug, and Cosmetic Act (2012c) 21 U.S.C. Section 360n: priority review to encourage treatments for tropical diseases
Federal Food, Drug, and Cosmetic Act (2012d) 21 U.S.C. Section 360(ff): priority review to encourage treatments for rare pediatric diseases
Federal Food, Drug, and Cosmetic Act (2012e) 21 U.S.C. Section 356(b): designation of a drug as a fast track product
Federal Food, Drug, and Cosmetic Act (2012f) 21 U.S.C. Section 356(a): designation of a drug as a breakthrough therapy
Federal Food, Drug, and Cosmetic Act (2012g) 21 U.S.C. Section 356(c): accelerated approval of a drug for a serious or life-threatening disease or condition, including a fast track product
Federal Food, Drug, and Cosmetic Act (2012h) 21 U.S.C. Section 355c: research into pediatric uses for drugs and biological products
Hsu J, Serrano D, Bhowmick T et al (2011) Enhanced endothelial delivery and biochemical effects of α-galactosidase by ICAM-1 targeted nanocarriers for Fabry disease. J Control Release 149:323–331
Maga AJ, Zhou J, Kambampati R et al (2013) Glycosylation-independent lysosomal targeting of acid α-glucosidase enhances muscle glycogen clearance in Pompe mice. J Biol Chem 288:1428–1438
Orphan Drug Act (1983), Pub. L. 97–414. 96 Stat. 2049. Amended in 1984 by Pub. L. 98–551 to add a numeric prevalence threshold to the definition of rare diseases
Ramsey BW, Davies J, McElvaney NG et al (2011) A CFTR potentiator in patients with cystic fibrosis and the G551D mutation. N Engl J Med 365:1663–1672
US Depart HHS FDA (2014) Guidance for industry: expedited programs for serious conditions--drugs and biologics. Retrieved August 20, 2014 from http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM358301.pdf
US Food and Drug Administration (2012a) Code of federal regulations title 21: drugs intended to treat life threatening and severely-debilitating illnesses. Pt. 312.80 (Subpart E). US Government Printing Office, Washington
US Food and Drug Administration (2012b) Code of federal regulations title 21: expanded access to investigational drugs for treatment use. Pt. 312.300 (Subpart I). US Government Printing Office. Washington
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Lacana, E., Yao, L., Pariser, A., Rosenberg, A., Woodcock, J. (2015). Building Biobetters: The Regulatory Landscape. In: Rosenberg, A., Demeule, B. (eds) Biobetters. AAPS Advances in the Pharmaceutical Sciences Series, vol 19. Springer, New York, NY. https://doi.org/10.1007/978-1-4939-2543-8_16
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DOI: https://doi.org/10.1007/978-1-4939-2543-8_16
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